Aquinnah has been awarded four peer-reviewed grants, two from the ALS Association, and one each from the National Institute of Health (National Institute of Neurological Disorders and Stroke) and the Massachusetts Life Science Center.  Our approach has also garnered interest and support from major pharmaceutical companies, including Takeda Pharmaceuticals.

The compounds Aquinnah discovered measure up against the most exacting standards: They are potent, orally bio-available in small doses, and can get past the blood brain barrier, a critical factor that hasn’t always been tested by others in the field.

 

We are currently conducting animal trials.  Human trials are scheduled to begin in the next 2-3 years. Once in human trials, we should know in just one year if the drug is effective because ALS is such a rapidly progressing disease.  Once the drug is proven successful in ALS patients, clinical trials will begin for Alzheimer’s disease.

Both Dr. Larsen and Dr. Wolozin are very optimistic about the future of neurodegenerative diseases. The discovery that the molecules in stress granules are the building blocks of neurodegenerative disease provides a new foundation for not just one, but potentially
many new therapies.

Historically, of all the branches of medicine, the study of neurology has the most unknowns and the fewest treatments, however, Dr. Wolozin believes that is about to change.  Scientists have been studying the molecular mechanisms of these diseases for 30 years.  It's important to remember that progress moves in increments.  For example, it took a long time to go from the invention of the telephone to the iPhone.  Drug development also takes time, but can make similar dramatic leaps forward.

 


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